New Uses For Old Antibiotics May Improve CF Treatment

New drug derivatives of antibiotics may be future treatment options for cystic fibrosis patients. Indiana University Research and Technology Corp. recently entered into an agreement with La Jolla Pharmaceutical Co. to develop “next-generation” offhoots of gentamicin, an antibiotic.

“We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives,” said Dr. George Tidmarsh, President and CEO of La Jolla, in a news release from Indiana University. “We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin but improve the therapeutic window, thereby potentially increasing the market opportunity as antimicrobial agents and potentially creating new opportunities for aminoglycosides in rare genetic disorders.”

Gentamicin has been used in hospitals to treat staph, urinary tract, heart, and pregnancy-related infections. However, it is highly toxic to the kidneys if used for a prolonged period of time. Since cystic fibrosis is a life-long disease that requires constant treatment, Dr. Bruce Molitoris at Indiana University’s Department of Medicine worked to discover less toxic derivatives of gentamicin.

Laboratory tests conducted to date show that the two developed compounds, named LJPC-30Sa and LJPC-30Sb, show biologic activity similar to gentamicin but have fewer toxic effects. This is ideal, because even though gentamicin shows proof-of-efficacy for patients with cystic fibrosis in short-term clinical tests, chronic treatment would be hindered by toxicity.

Now that La Jolla has the rights to LJPC-30Sa and LJPC-30Sb, the company will further develop the compounds to treat cystic fibrosis and other serious bacterial infections. They are also looking to expand to other genetic disorders such as Duchenne muscular dystrophy. Researchers and faculty at Indiana University Research and Technology Corp. will continue in their efforts to develop groundbreaking new treatments for diseases such as cystic fibrosis.

If successful in developing less toxic forms of gentamicin, La Jolla would enable enhanced therapies for the 70,000 patients with cystic fibrosis in the world.

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